Patent SG-11201909203W-A (SG)
Title: Tissue selective transgene expression
Inventor: TAGLIATELA STEPHANIE
IPC: A01K67/027
Filed: 2018-04-03
Abstract: 1 N co 00 O O (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 11 October 2018 (11.10.2018) WIPO I PCT omit °nolo mol mom!! m Immo oimIE (10) International Publication Number WO 2018/187363 Al (51) International Patent Classification: C12N 15/63 (2006.01) C12Q 1/68 (2018.01) AO1K 67/027 (2006.01) (21) International Application Number: PCT/US2018/025940 (22) International Filing Date: 03 April 2018 (03.04.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/480,998 03 April 2017 (03.04.2017) US (71) Applicant: ENCODED THERAPEUTICS, INC. [US/US]; 343 Oyster Point Blvd., Suite 100, South San Francisco, California 94080 (US). (72) Inventors: TAGLIATELA, Stephanie; 343 Oyster Point Blvd., Suite 100, South San Francisco, California 94080 (US). YOUNG, Andrew; 343 Oyster Point Blvd., Suite 100, South San Francisco, California 94080 (US). CHEN, Szu-Ying; 343 Oyster Point Blvd., Suite 100, South San Francisco, California 94080 (US). RAMAMOORTHI, Kartik; 343 Oyster Point Blvd., Suite 100, South San Fran- cisco, California 94080 (US). OBERKOFLER, David; 343 Oyster Point Blvd., Suite 100, South San Francisco, California 94080 (US). (74) Agent: YIN, Eva F. et al.; WILSON SONSINI GOODRICH & ROSATI, 650 Page Mill Road, Palo Alto, California 94304-1050 (US). (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, (54) Title: TISSUE SELECTIVE TRANSGENE EXPRESSION FIG. 4B S EQ D NO: 8 SEQ ID NO: 1 EFla (57) : Provided herein are compositions and methods for selective expression of a transgene. Compositions and methods for selective expression of a transgene comprise one or more human regulatory elements, which, when operably linked to a transgene, can facilitate selective expression of a transgene (e.g., cell-type selective expression) in a target cell as compared to at least one or more non-target cells. [Continued on next page] WO 2018/187363 Al MIDEDIMOMOIDEIREEMOMOHOHIIMIMMOVOIMIE GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Declarations under Rule 4.17: as to the applicant's entitlement to claim the priority of the earlier application (Rule 4.17(iii)) Published: with international search report (Art. 21(3)) before the expiration of the time limit for amending the claims and to be republished in the event of receipt of amendments (Rule 48.2(h)) with sequence listing part of description (Rule 5.2(a))